Breakthrough Therapy Designation

The Food and Drug Administration Safety and Innovation Act (FDASIA), signed into law on July 9, 2012, introduced several reforms that are intended to streamline the FDA's premarketing approval process for drugs and devices.

Among these reforms was the creation of a new expedited development pathway for drugs that are intended to treat serious or life­-threatening conditions.

Under this pathway, a drug candidate may be designated by the FDA as a "breakthrough therapy" if early clinical evidence indicates that the drug may demon­strate a substantial improvement over existing therapies. Once a drug has been granted this status, the FDA commits to working particularly closely with the drug's sponsor to create an efficient development plan and facilitate its approval.

The full impact of the breakthrough therapy designation will not be clear for some time, but efforts are under way to address some of the issues described in this brief.